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Thursday, May 29, 2008
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12:00p.m. to 3:00p.m.
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Conference Registration
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3:00 p.m. to 3:10p.m.
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Welcome, Greg Macres (CGRF)
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Session 1. Basic science – pathological mechanisms. Chairs: Tony Futerman Ph.D.
and Col. Kondi Wong M.D.
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3:10p.m. to 3:30p.m.
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“Overview of basic science session” Tony Futerman Ph.D. (Weizmann Institute of
Science – Rehovot – Israel)
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3:30 p.m. to 4:15 p.m.
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“Molecular Mechanisms of Lysosome Biogenesis” Juan S. Bonifacino, Ph. D. - National
Institutes of Health - Bethesda, Maryland
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4:15. p.m to 5:00 p.m.
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“Fate and function of glucosylceramide in mammalian cells” Gerrit van Meer, Ph.
D. - Utrecht University - The Netherlands
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5:00 p.m. to 5:20 p.m
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“The role of defective calcium-homeostasis in the neuropathology of type 2 and 3
Gaucher disease”. Einat Vitner, Ms.c (Weizmann Institute of Science - Rehovot
– Israel)
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5:20p.m. to 5:35 p.m.
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TBD: Selected from Poster Board Abstracts
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6:30p.m. to 9:00p.m.
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Cocktail Reception and Dinner - Poolside
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Friday May 30, 2008
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7:00a.m. to 8:00a.m.
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Continental Breakfast
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8:00a.m. to 8:05a.m.
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Opening Remarks
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Session 1. (Continued) Basic science – pathological mechanisms. Chairs: Tony Futerman
Ph.D. and Col. Kondi Wong M.D.
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8:05a.m. to 8:50a.m.
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“Glucosylcerebroside accumulation in gba2 knockout mouse” Yildiz Yildiz, M.D.
- University of Bonn, Bonn - Germany
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8:50a.m. to 9:35a.m.
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Novel metabolic pathway of glucosylceramide involving Klotho-related protein: Insights
into Gaucher disease Makoto Ito, Ph. D. - Kyushu University - Japan
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9.35a.m. to 9.50
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TBD: Selected from Poster Board Abstracts
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9:50a.m. to 10:20a.m.
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Coffee Break
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10.20a.m. to 11.05a.m.
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“Autophagy in the pathophysiology of the brain”
Ana Maria Cuervo M.D. Ph.D – Albert Einstein College of Medicine – Bronx – New York
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11.05a.m. to 11.50a.m.
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“GM1-ganglioside as apoptotic signal in ER- and mitochondria-mediated cell death
in neurodegenerative GM1 gangliosidosis” Alessandra d'Azzo , Ph.D. (St. Jude Children’s
Research Hospital - Memphis, Tennessee
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11.50a.m. to 12.05p.m.
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TBD: Selected from Poster Board Abstracts
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12:05p.m. to 12:50p.m.
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Lunch
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12:50p.m. to 1:35p.m.
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“Common themes in Batten disease pathogenesis” Jonathon Cooper, Ph. D. - King's
College – London
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1.35p.m. to 1.50p.m.
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TBD: Selected from Poster Board Abstracts
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1:50p.m. to 2.30p.m.
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Basic Science – Panel Discussion With all Presenters – Q&A Moderators: Tony Futerman
Ph.D. and Col. Kondi Wong M.D.
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2:30p.m. to 3:15p.m.
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Gaucher disease and Parkinsonism Ellen Sidransky, M.D. – National Institutes of Health – Bethesda, Maryland
Moderator: Gregory A. Grabowski, M.D.
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3:15p.m. to 5:15p.m.
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Poster Session
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6:00p.m. to 9:00p.m.
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Cocktail Reception/Dinner
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9:00p.m.
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Desert Wine Served on the Veranda
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Saturday, May 31, 2008
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7:30a.m. to 8:30a.m.
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Continental Breakfast
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8:30a.m. to 8:45a.m.
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Opening Remarks and overview Gregory A. Grabowski, M.D.
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8:45a.m. to 9:30a.m.
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“Therapeutic trials and pathogenetic studies in an animal model of metachromatic
leukodystrophy” Volkmar Gieselmann M.D. - University of Bonn - Bonn, Germany
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9:30a.m. to 10:15a.m.
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“New therapeutic approaches for lysosomal disorders of the brain” Fran Platt,
Ph. D. - University of Oxford, United Kingdom
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10:15a.m. to 10:45a.m.
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Coffee Break
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10:45a.m. to 11:30a.m.
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“Prospects for Treating the CNS Disease in LSDs” Seng H. Cheng , Ph. D. - Genzyme
Corporation - Framingham, Massachusetts
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11:30a.m. to 12:15p.m.
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“Re-Engineering Lysosomal Enzymes for Intravenous Delivery to the Human Brain”
William M. Pardridge, M.D. (Bill) - University of California at Los Angeles - Los
Angeles, California
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12.15p.m. to 12.30p.m
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TBD: Selected from Poster Board Abstracts
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12:30p.m. to 1:15p.m.
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Lunch
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1:15 p.m. to 2:00p.m.
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“Pharmacological chaperones as a potential therapy for lysosomal disorders of the
brain” Brandon A. Wustman, Ph. D. - Amicus Therapeutics – Cranbury – NJ
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2:00p.m. to 2:15p.m.
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TBD: Selected from Poster Board Abstracts
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2:15p.m. to 3:00p.m.
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Therapy – Panel Discussion With all Presenters – Q&A Moderators: Roscoe Brady
M.D. and Raphael Schiffmann M.D.
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